Dr. Charles Macias (Texas Children's Hospital) talks about the inception, challenges, and logistics of a large Research Network, the PEMCRC (Pediatric Emergency Medicine Collaborative Research Committee).

1. POISE Network Webinar
October 25, 2010
Charles G Macias MD, MPH
Baylor College of Medicine- Texas Children’s Hospital
Houston, Texas
Past Chairman, PEMCRC

2. Objectives
• To discuss the benefits to collaborative research through
research networks
• To describe barriers to effective performance of research
networks
• To discuss obstacles and solutions noted in critical
analyses:
– Pediatric Clinical Research Networks (PCRNs)
– Inventory and Evaluation of Clinical Research Networks (IECRN)
• To summarize translation of key points through practice
examples in the Pediatric Emergency Medicine
Collaborative Research Committee (PEMCRC)

3. Challenges of pediatric research
• Low mortality, low morbidity
– Larger sample sizes
• Pediatric under-representation in literature
– Fewer valid and reliable tools
– Timeliness and efficiency barriers with single
institutions
• Limited funding

4. Benefits of collaborative research
• Increases opportunities to achieve sample sizes
in shorter time frames
• Allows for diversity in representation of
populations (generalizability)
– Geographic areas
– Etiologies of infectious diseases
– Mix of ethnicity and race
• Successes of PECARN and PEMCRC

5. Pediatric Clinical Research Networks
• AAP/PROS assessment
– Internet search
– 21 interviews with network leaders
– Survey of 43 AAP leaders
• 67 PCRNs
– 40% are specialty networks
– Specialty: more publishable, primary-care most adept
at launching

6. What works well?
• Well-developed base of expertise in study design,
feasibility assessment, protocol review, and feedback on
applying protocols to specific populations
• Importance of shared vision and defined mission
• Core infrastructure (human resources, funding,
information technology) and affiliations with larger
professional associations or institutions
• Sense of research culture and core values within a
subspecialty
• Expertise located in data coordinating centers

7. What works poorly?
Network management/project management
• Setting research priorities
• Sustaining network infrastructure with limited funding
• Maintaining site interest and enthusiasm over time
• Managing change and growth
• Finding ways to partner with other networks and
disciplines
• Finding ways to support face to face meetings

8. Project management concerns
• Protocol complexity, time for patient accrual and staff
effort tasks
• Disconnects between budgets and site
demands/workloads
• Logistics in coordination of IRB approvals, contracts,
business agreements and regulatory compliance
• Paucity of qualified research assistance and rate of
turnover
• Efficient data management and quality check systems
lacking

9. Role of Clinical and Translational Science Award
• Limited role of pediatric demands for CTSA
involvement
• PCRNs are challenged to influence national
politics of the CTSA program
• National Center for Research Resources
(NCRR) could aim pediatric strategies at PCRNs

10. Data collection
• Forms:
– Paper/pencil
– Hardcopy worksheets to electronic
– Electronic options:
• Teleform™
• Registries
• Web: Access™
• Variability of EMR/EHR:
– Extraction vs systems reporting
– Embedding protocols
• Data center mechanisms for feedback to sites
within a network for integrity of quality

11. Best practices
• Sharing of best practices is itself a best practice
• Connecting special interests
• Sharing structures with other PCRNs
– Governance models
– Industry relationships
– Communication strategies
– Data collection methodologies
– Training strategies
• Face to face conferences for sharing (QI)

12. Societies and organizations
• Support and recognize network researchers
• Facilitate network research resources locally
• Promote research networks for AAP guideline
development
• Link PRCN research to ABP Maintenance of
Certification

13. PCRNs key issues
• Creating a learning system
• Regulatory efforts
• Awareness of current climate of cost-shifting and
cross-subsidization (resources)
• Lack of a business model
• Challenges of EMRs and EHRs
• Lack of clarity in the CTSA role

14. Assessment and strategic planning
• What is currently being done?
• What could reasonably be done with resources?
• What could be done to expand resources?
• What strategies could be helped by interaction
with other networks?
• Wikified utility for resources among networks

15. Inventory and Evaluation of Clinical
Research Networks (IECRN)
• Commissioned by the NIH Roadmap for Medical
Research in the 21st
Century
• Overall goal of translating basic sciences into
more usable clinical activities by making clinical
networks more efficient and effective

16. IECRN objectives
• The four major objectives of IECRN were to:
– Develop an inventory and database of clinical research networks
– Conduct a survey of a subset of clinical research networks to
provide a detailed description of current practices used by
networks to accomplish their goals, as well as barriers and
facilitators to their success
– Identify networks that have had particular successes and study
the practices that have contributed to that success
– Conduct a National Leadership Forum to present and discuss
the practices that are identified as possible best practices for
network achievement

17. Core survey: qualitative component
• 262 research networks met core methodology
definition for inclusion
• 95% response rate to survey
• Network age range:
– 6-50 months (median of 6 yrs)
• NIH funded networks conducted a greater
percentage of clinical trials

18. Key findings
• A network must have strong and committed
investigators
– Expertise and involvement
– Clarity of scientific focus
– Practice based network participants must have
commitment to be open to new knowledge
– Sponsor commitment and vision if relevant

19. Key findings
• Openness to new participants
– Prevents the network from becoming stagnant
• New ideas and new energy
– Invite outside scientists (build inter-organizational
relationships)
– Young participants require mentoring and support

20. Key findings
• Importance of time
– Reduced grant writing time vs increased participation
time
– Lack of protected time (developing vs enrolling)
– Lack of time set aside by networks for analysis and
writing of manuscripts

21. Key findings
• Lack of funding
– Limits scope of activity
• Chronologic decision-making: setting an agenda and
searching for funding afterwards
– Lack of flexibility in how money can be spent: multiple
sites increases complexity of budgets
– Support of existing or expanding infrastructure
• Strategies to adapt to limited funds: ex simplicity rather than
complex assessment methods or data collection methods
• vs searching for external infrastructure support (ex.
pharmaceutical)

22. Key findings
• Uncertainty of core support
– Need for certainty of core infrastructure is critical to
appeasing the need to enhance organizational
capability of individual sites
– Lack of funding for core support staff

23. Key findings
• Tension between collaboration and competition
– Need leadership in young investigators
– Need opportunities for young investigators to become
first authors on network projects
– Process to ensure effective communication
• Regularly scheduled communication venues
• Conferences to enhance skill sets
– Structure of guidelines, processes, and timelines

24. Key findings
• Incentives for cross network collaborations
– Commonality of interests with other
networks/investigators
– Build on existing foundations, partnerships, or
societies
– This strategy is rarely within a network’s budget

25. Key findings
• Establishing buy-in for network research
– A network’s productivity is dependent on buy-in from
its stakeholders
• Level of engagement for agenda setting
• Level of engagement for voting/prioritizing/strategic planning
– Feasibility of studies: are there subgroups to assess?
(ex. PECARN, Community advisory groups)
– Staff buy-in for training

26. Key findings
• Bureaucratic and regulatory hurdles
– Variations in IRB functioning
– Understanding of regulatory activities (ex. data
transfer)
– Reviews by sponsors who may not understand the
network goals
– Operating procedures for the network and the site
• Manuals of operations
• Policies and procedures

27. Key findings
• Well-trained and appropriate staff
– Administrative support for network
– Selection of centralized tasks for network
• Ex. data quality assessments
– Staff turnover and strategies to assure longevity
• Minimize variation in training
– Training and professional development of staff
• Strength in academically affiliated institutions

28. Key findings
• Use of information technology
– Need well integrated systems compatible across the
network
– Focus on modular tools that are not system
dependent
– Centralization of software as a strategy
– Standardization of protocols/data elements within
systems
– Webcast tools/learning systems and communication
– Electronic data capture to minimize human effort
– Appropriate use: avoid using technology for
technology sake

29. Key findings
• Value of standardization
– Using protocol templates
– Extensive and robust SOPs within manuals
– Barriers to standardization are time related: coming to
consensus

30. Key findings
• Access to participant populations
– Careful site evaluation and selection
– Retention of participants: sharing best strategies
– Transparency: monitoring of recruitment and retention
• Blinded report cards
• Healthy competition among sites

31. Key findings
• Dissemination of key results
– Top down approach: publication of results by
academic centers with passive uptake
– Bottom up approach: directly influence practitioners
– Access large institutional providers with a single
organizational structure for multiple entities (ex. VA
research networks)

32. PEMCRC Network: stakeholders
• A network of volunteer researchers affiliated
through membership in the American Academy
of Pediatrics
– 159 active members
– Represent 52 academic institutions
– 20 institutions have infrastructures to potentiate
implementation of almost all PEMCRC studies

34. Active research collaboratives
Study
Collaborative
Number of
Investigators
Number of
Institutions
Number
of
patients
Number of
publications
Additional
substudies
Meningitis 24 20 3295 4+1 4+1
SBI in infants 11 8 1248 4 5
UTI 30 19 2477 3 abstracts 3 planned
Appendicitis 19 10 ? (2000 in
Dec) of
2500
Data
collection
closed April
2 planned
MgS04 asthma
survey
10 8 324 1 abstract
1 submission
0
POISE 25 25 527
(interns)
0 Network
H1N1 PERN Study 99 99 ? 1 0

35. Challenges
• Permanent infrastructures and infrastructure funding
– Iterative process of self-definition based upon:
• Skills sets of members
• Industry
• Growth of PECARN and maturing of federal recognition for PEM
research
• Availability of funding
• Strength of the AAP: people and money
• Young investigators, mentorship for longevity of the
network
• 3 year leadership roles- building institutional memory

36. Network: support
• Limited administrative support
– Monthly teleconferences for each study collaborative-AAP
– AAP supports venues for biannual meetings but individuals
participate with their own resources
– No permanent infrastructure for patient enrollment or chart
review: no research coordinator support
– Research collaboratives bring their own funding where
necessary
• Data center
– Data processing and data quality support centralized
– Contributed personnel support (informatician, biostatistician)
– Projects
» UTI collaborative
» Appendicitis collaborative
» H1N1 PERN study

37. Administrative achievements
• Productivity based upon volunteerism
– Strength of the drive of PEM researchers
– Evolution of efficiencies and expertise in research
• Development of an increased number of mentors for support
» PECARN researchers/mentors
» EMSC/NEDARC partnership- young investigator grant
writing scholarship
• Evolving partnership with pharmaceutical
industry
• Formalization of a survey review mechanism
separate from study review process

38. The future
• Enhancements of virtual collaboratives
– www.pemcrc.org
– Migrating the AAP SOEM network to www.pemnetwork.com
– Enhancement of the network will allow subsites for
www.pemcrc.org to create
• Wikified sites for active protocol development for each collaborative
• Multi-media for virtual training
• Shared compliance document libraries
• Web/pod casts

39. Key messages for collaborative research
• Support the networking of people with a shared
vision/mission
– Expand the talents of stakeholders with expertise in study design
through education and mentorship
– Identify champions at each site (may not be the investigator) and
assure effective communication strategies are in place
– Encourage new participants in effective and meaningful way
• Centralize administrative functions as much as possible
(DUAs, protocols)
– Clear governance descriptions
• Standardize
– Consider reuse of tools to maximize familiarity (SOPs)
– Develop strategies for effective use of EMR

40. Key messages for collaborative research
• Provide sharing of models for protected time
• Create and enpower workgroups with enough structure
and timeline guidance to be effective
– Include grant writing groups for projects and for core
infrastructure support
– Staff training module development
• Encourage well-defined links to professional societies,
organizations and industry
• Develop mechanisms for transparency in enrollment
• Create detailed dissemination strategies and consider
expansion into knowledge translation research
• Collaborate with other networks for economies of scale

41. Collaboration among networks
-H1N1 Case control study
99 centers worldwide

42. References
• IECRN (Inventory and Evaluation of Clinical Research
Networks), Complete Project Report, 2006
• Pediatric Clinical Research Networks: Optimizing
Effectiveness through Cooperation, Proceeding notes,
2009
• Klassen TP, Ackworth J, Bialy L, et al. Pediatric
Emergency Research Networks: a global initiative in
pediatric emergency medicine. Pediatr Emerg Care.
2010; 26(8):541-3.